Under a new Medicaid-focused Innovation Center model, the Centers for Medicare and Medicaid Services (CMS) will encourage agreements between pharmaceutical companies and state Medicaid agencies for cell and gene therapies (CGT) targeting sickle cell disease (SCD).
The Centers for Disease Control and Prevention (CDC) estimates that approximately 100,000 Americans have sickle cell disease. About half are enrolled in Medicaid and 11% are enrolled in Medicare, often as dually eligible enrollees in both Medicaid and Medicare, according to CMS.
Cell and gene therapy reimbursement is a challenging issue. These treatments can cost $1 million or more per dose, leaving the companies that develop them looking for new payment models and financing arrangements. One avenue might be the National Heart, Lung, Blood Institute (NHLBI) Cure Sickle Cell Initiative (CureSCi), which has been underway since 2018.
Clinical research over next five years
NHLBI CureSci seeks to bring cell and gene therapy into clinical research with gusto between 2023 and 2028. As a result, millions in grant funds have been made available. Academic researchers, private sector researchers, and patients should be able to expect a variety of opportunities.
“The Cure Sickle Cell Initiative aims to develop curative strategies by initially focusing on genetic therapies that modify hematopoietic stem cells (HSCs), which make red and other blood cells," the NHLBI explained. "These modified HSCs can then be used in bone marrow transplants." Progress would use genetic therapies to modify patients’ HSCs so that their red blood cells are no longer sickle-shaped. This might be accomplished by:
- Introducing the normal hemoglobin gene into the HSCs.
- Correcting the mutation in the abnormal hemoglobin gene and transferring that corrected gene into the HSCs.
- Modifying the HSCs so they stop making abnormal hemoglobin and start making other hemoglobin.
4 areas of CMS sickle cell action plan
- Expanding coverage and access
- Improving quality and the continuum of care
- Advancing equity and engagement
- Examining data and analytics
“Through the upcoming Cell and Gene Therapy (CGT) Access model, the CMS Innovation Center will test a new approach for administering outcomes-based agreements to help Medicaid enrollees,” stated CMS administrator Chiquita Brooks-LaSure in a recent blog post. “Specifically, the Model aims to establish a partnership among CMS, pharmaceutical manufacturers, and state Medicaid agencies." The model will evaluate multiple conditions including gene therapies for illnesses like SCD.
FDA cell and gene therapy decisions afoot
Cell and gene therapy-related research and development in the U.S. continues to grow at a fast rate, according to the U.S. Food and Drug Administration (FDA). Although only a few treatments have obtained FDA approval, many of these products are still advancing in clinical development. The Center for Biologics Evaluation and Research (CBER) regulates cellular therapy products, human gene therapy products, and certain devices related to cell and gene therapy.
Over the past 30 years, numerous companies have made moves in research and development of new techniques and technologies for cell and gene therapy. Companies such as Bluebird Bio, Bristol-Myers Squibb, BioMarin Pharmaceutical, Gilead Sciences, Orchard Therapeutics are among those that have invested many millions in new facilities and scientifics intended to build capacity for cell and gene therapy healthcare services. A majority of gene therapy drugs, for example, are injection-type treatments. This year was projected to be a major launch year, according to a CVS Health gene therapy report.
CMS plans to structure and coordinate multistate outcomes-based agreements (OBAs) with participating manufacturers on behalf of states. CMS would also take on the responsibility of implementing, monitoring, reconciling, and evaluating the financial and clinical outcomes outlined in these outcomes-based agreements (OBAs).
“It is going to be interesting to see what happens because they [gene therapies] are very expensive,” Aimee Tharaldson, PharmD, told Managed Healthcare Executive last year. “Some of them are coming to market with pretty good data and some will likely come to market with not the best data. They are just going to need to be evaluated carefully to make sure they are effective and safe and have a durable effect.”